A MIXED-METHODS STUDY TO EXPLORE THE HEALTH-RELATED QUALITY OF LIFE IMPACT ON CAREGIVERS OF CHILDREN WITH HEREDITARY ANGIOEDEMA
Author(s)
Ellen Howard, PhD1, Adam Gordois, MSc2, Katie Lofthouse, PhD1, Sandra Nestler-Parr, MPhil, MSc, PhD2, Patrick Gillard, PharmD2, Kate Williams, BSc, MSc, PhD1;
1Acaster Lloyd, London, United Kingdom, 2Biocryst Pharmaceuticals, Durham, NC, USA
1Acaster Lloyd, London, United Kingdom, 2Biocryst Pharmaceuticals, Durham, NC, USA
OBJECTIVES: Hereditary angioedema (HAE) is characterized by recurrent localized swelling episodes in the subcutaneous tissue (HAE attacks). HAE can adversely impact the health-related quality of life (HRQoL) of patients and their caregivers. This study aims to evaluate the HRQoL of caregivers of children with HAE and assess how a child’s HAE attack frequency affects caregiver wellbeing.
METHODS: Mixed-methods study with caregivers of children (aged ≥2 to <12 years old) with HAE, including a quantitative online survey and a qualitative interview. Caregivers were recruited through HAE patient organizations in the United States of America, the United Kingdom and Canada. The survey included demographic and clinical questions, the EQ-5D-5L, and the Pediatric Quality of Life Inventory-Family Impact Module (PedsQL-FIM). Caregivers completed the survey for the past three months, and two earlier three-month periods when their child had the most and least HAE attacks. Interview questions were open-ended to explore the impact of caring on participants’ daily lives during these periods. Survey data were analyzed descriptively; qualitative interview data were analyzed using thematic and content analysis. Findings will be synthesized using mixed-methods analysis. The results presented below are pending completion of recruitment in January 2026.
RESULTS: Thirteen caregivers (all parents) have completed the survey and interview. These participants reported a mean of 20.6 hours/week spent caregiving. Caregiving responsibilities included monitoring symptoms, managing medication and medical appointments, supporting daily activities, and providing emotional support. Caregivers reported a range of impacts on their daily lives, including exhaustion and lack of sleep; difficulty performing household, work, leisure, and social activities; and emotional strain. Caregivers experienced greater impacts on their HRQoL when their child had more frequent attacks.
CONCLUSIONS: Study findings highlight the substantial impact of caring for a child with HAE, particularly during periods when attacks occur more frequently.
METHODS: Mixed-methods study with caregivers of children (aged ≥2 to <12 years old) with HAE, including a quantitative online survey and a qualitative interview. Caregivers were recruited through HAE patient organizations in the United States of America, the United Kingdom and Canada. The survey included demographic and clinical questions, the EQ-5D-5L, and the Pediatric Quality of Life Inventory-Family Impact Module (PedsQL-FIM). Caregivers completed the survey for the past three months, and two earlier three-month periods when their child had the most and least HAE attacks. Interview questions were open-ended to explore the impact of caring on participants’ daily lives during these periods. Survey data were analyzed descriptively; qualitative interview data were analyzed using thematic and content analysis. Findings will be synthesized using mixed-methods analysis. The results presented below are pending completion of recruitment in January 2026.
RESULTS: Thirteen caregivers (all parents) have completed the survey and interview. These participants reported a mean of 20.6 hours/week spent caregiving. Caregiving responsibilities included monitoring symptoms, managing medication and medical appointments, supporting daily activities, and providing emotional support. Caregivers reported a range of impacts on their daily lives, including exhaustion and lack of sleep; difficulty performing household, work, leisure, and social activities; and emotional strain. Caregivers experienced greater impacts on their HRQoL when their child had more frequent attacks.
CONCLUSIONS: Study findings highlight the substantial impact of caring for a child with HAE, particularly during periods when attacks occur more frequently.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR61
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Pediatrics, SDC: Rare & Orphan Diseases